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Nat Rev Genet. 2011 May;12(5):301-15. doi: 10.1038/nrg2985. Epub 2011 Mar 29.

Ex vivo gene transfer and correction for cell-based therapies.

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HSR-TIGET, San Raffaele Telethon Institute for Gene Therapy and Vita Salute San Raffaele University, San Raffaele Scientific Institute, via Olgettina 58, 20132 Milan, Italy.


Cell-based therapies are fast-growing forms of personalized medicine that make use of the steady advances in stem cell manipulation and gene transfer technologies. In this Review, I highlight the latest developments and the crucial challenges for this field, with an emphasis on haematopoietic stem cell gene therapy, which is taken as a representative example given its advanced clinical translation. New technologies for gene correction and targeted integration promise to overcome some of the main hurdles that have long prevented progress in this field. As these approaches marry with our growing capacity for genetic reprogramming of mammalian cells, they may fulfil the promise of safe and effective therapies for currently untreatable diseases.

[Indexed for MEDLINE]

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